Whose Data Are They Anyway? Can a Patient Perspective Advance the Data-Sharing Debate? – NEJM Perspective

Most patients haven’t thought much about data sharing, according to Sara Riggare, but those who have “find the current system unreasonable. Patients expect that health care professionals and researchers use patient data in the best possible way. That there is a fight over what the best way is is perplexing and disappointing.”

Riggare is an engineer and doctoral student at the Health Informatics Center at Karolinska Institutet in Stockholm, where she researches models and methods for “digital self-care” in chronic disease — ways to use technology in monitoring and treating oneself. She is also a patient. Riggare had her first symptoms of Parkinson’s disease in her early teens and calls herself a “digital patient.” Actively engaged in her own care, she advocates both for patients’ right to access their own medical data and for the health care system to more actively use patients’ experiences as a resource. She shares her opinions on her blog, “Sara. Not patient but im-patient.”1

Like Riggare, the patients who participated in the recent Journal summit on aligning incentives for data sharing want their data shared quickly, especially to ensure that other patients know about possible side effects. But they also want some control over how the data are shared. For example, they would be more hesitant to participate if commercial or other interests were involved — for instance, if health care systems wanted to use the data to decide whether to provide care to certain groups or if drug or insurance companies had a commercial interest in them.

Patients also said they wanted trial results to be shared with participants themselves, along with an explanation of what the results mean for them — something that generally doesn’t happen now. In addition, most participants said they had entered a trial not to advance knowledge, but to obtain what their doctor thought was the best treatment option for them. All of which raises some questions about how well patients understand consent forms.

To some extent, what informed consent means to the parties involved guides thinking about who owns and should control clinical trial data. The value of trials for society and other patients is not necessarily the same as what benefits individual participants in research projects. Moreover, participants may be vulnerable and dependent on their doctor’s advice and follow-up. They may feel pressured to participate or may not understand the full implications of doing so — all of which contributes to the ethical dilemmas that necessitate careful regulation of medical research.

Before World War II, there were no written international rules or conventions regulating medical research. The concept that “the voluntary consent of the human subject is absolutely essential” was introduced in the Nuremberg Code,2 which was formulated in August 1947 by American judges presiding over the trial of Nazi doctors accused of conducting murderous and torturous human experiments in concentration camps.

It gradually became clear that the research community needed to further intensify its ethical awareness. For example, “voluntary consent” was not a license to conduct unethical experiments or do whatever the researcher wanted with patient data. The World Medical Association coordinated the work leading up to the Declaration of Helsinki, adopted in June 1964 (and since revised seven times). Though the Declaration isn’t legally binding, it has been codified in or influenced much national and regional legislation.

In 2008, the U.S. Food and Drug Administration ceased requiring trials conducted outside the United States to comply with the Declaration, and U.S. and European legislation now seems to be headed in different directions regarding data sharing. Simply put, the disagreement is over whether “consent” means that the patient transfers property rights to researchers or that patients own the data and give researchers license to use them for a defined purpose.

One summit attendee, Sharon Terry — president and CEO of Genetic Alliance, a network that aims to help people take charge of their own health, and the mother of two children with a rare genetic disorder — remarked that “Trial participants are not patients in the traditional sense of the word. It really should be looked at as a partnership.”

Yet the discussion about data sharing has largely taken place between clinical trialists, who spend years collecting, curating, and analyzing data from clinical trials, and data scientists, who would like to add value to those data by reanalyzing and reusing them in novel ways. The discussion has been about data ownership, whether a reanalysis conducted independently of the original research group will ever be valid, and whether it’s fair for data scientists to get data “for free.” Both sides claim to have the patient’s and the public’s best interests at heart, but not many partisans of either camp have asked patients what those interests are.

Patients and patient representatives were grateful to have a seat at the summit table and provided fresh perspectives on research, medicine, and patient care. “Digital patients” like Riggare and Dave deBronkart (who calls himself “e-patient Dave”), public advocates like Terry, and the creators and users of initiatives such as PatientsLikeMe and Quantified Self don’t want to be passive observers and sources of research data. They use the power of the Internet to engage in their own care, interact with clinicians and fellow patients, create new knowledge, and suggest new ways of delivering health care. They believe in sharing data and experiences in order to help themselves and fellow patients. Anna McCollister-Slipp, chief advocate for participatory research at the Scripps Translational Science Institute, reminded the summit audience that “Patients are smart, very receptive — and many of us have day jobs that can bring a whole new set of skills to medical research. We know how it is living with disease and can help solve problems faster.”

Asked whether she worried about patient privacy in the event that trial data became widely shared, Riggare said the need for “privacy is not a constant. It varies depending on the context. If you have a life-threatening disease and need help, you do not care much about privacy. The question is also: How many people will die if we don’t share data? Personally, I know people whose lives have been saved because tumor data were shared.”

Riggare’s point is that receiving health care always implies a loss of privacy. Patients must expose personal information to get help, and that help is usually built on knowledge gained from experiences of previous patients who have revealed personal information. Patients want their data used responsibly, however, so the question is really: Who should control how data are distributed and used by others? The patients themselves? Doctors and researchers? Research institutions or governments?

Traditionally, doctors collected and protected patients’ health information. As health care has become more complex and information has been shared between doctors and other health care workers, among institutions, and sometimes between countries, the legal and ethical framework securing that information has also grown increasingly complex. In addition, laws vary from country to country. This complexity and variation hinder sharing of patient data, whether from clinical trials or electronic health records.

In the United States, for example, absent specific language to the contrary in informed consent documents, research participants don’t have to give specific permission for their deidentified data to be used by other researchers. Indeed, the courts have ruled that even biospecimens don’t belong to patients. Europe seems to be moving in the opposite direction — requiring explicit consent for reuse of data or data sharing and allowing patients to withdraw their consent at any time. The relevant legislation is expected to be strengthened next year.3 Other regions will have their own concerns and priorities, but it seems unlikely that many will force or encourage their people to give up rights to their own clinical data if they participate in research.

Perhaps the solution to the data-sharing struggle lies in shifting data ownership and control to individual patients everywhere. What such a policy would look like in practice remains unclear, but current technology makes it easy to stay in touch with trial participants and seek their renewed consent, and dynamic consent forms are being developed and tested.4 Seeing clinical trial data as the property of each patient might simplify the data-sharing discussion: the patient shares, first with the clinical trialists and then, if the patient wishes, with data scientists.